NICE Recommends Against BioMarin’s Drug for Very Rare Metabolic Disorder

The UK’s National Institute for Health and Care Excellence (NICE) has recommended that the National Health Service (NHS) not offer BioMarin Pharmaceutical’s Vimizim (elosulfase alfa) for treating mucopolysaccharidosis type 4A syndrome, also known as Morquio A syndrome.

The UK cost regulator said it couldn’t justify the drug’s price, which is over $1,000 for a 5-mg vial, and that the company’s cost modeling wasn’t “robust” or “plausible” because it didn’t adequately capture disease progression for the rare, life-limiting metabolic condition.

“We are disappointed not to be able to recommend elosulfase alfa for use within the NHS,” said Meindert Boysen, NICE’s deputy chief executive and director of the Centre for Health Technology Evaluation, who noted that the “length of the managed access agreement has been extended twice to allow the company more time for its submission.”

Still, NICE said the therapy’s trial data showed some long-term benefits suggesting it could help stabilize the disorder in patients. And Boysen pledged to continue working with BioMarin to try and reach an agreement ahead of the next committee meeting in January 2022.